Worldwide access for children to sight-saving medication

The eye disease uveitis affects thousands of children every year - but a landmark trial has enabled worldwide access to new, sight-saving medication.

Prior to the Uveitis (Sycamore) Study, there were few evidence-based therapies for children with moderate to severe uveitis, leading to many suffering serious visual complications and often blindness.

Uveitis: a potentially blindness-inducing disease

Approximately 1 in 1000 children in the UK develop JIA (Juvenile Idiopathic Arthritis). Both boys and girls are affected, and of those children with JIA, around 30-40% are at risk of inflammation of the uvea in the eye, known as uveitis. In one third of the children who develop uveitis, the disease is sufficiently severe as to cause visual loss, cataracts, increased pressure in the eye and blindness.

This is partly due to the level of damage in the eye already present when the diagnosis is made. The existing treatments did not control the uveitis or JIA but reduced inflammation, so relieving symptoms and reducing the level of permanent damage. However, even with these, the majority of children with moderate to severe disease continued to have active inflammation in the eye, leading to such loss of vision in one eye that they were certified blind as a result of the uncontrolled inflammation.

A landmark trial success

Professor Athimalaipet Ramanan’s research focuses on paediatric rheumatology, and has involved him in a number of controlled trials of novel biological drugs in children with JIA associated uveitis. He also leads on new agents for management of refractory arthritis in collaboration with industry, devising and conducting studies in rare disease using novel study designs.

Pharmaceutical company AbbVie Inc had developed the drug Adalimumab, which was approved for treatment of childhood arthritis in the UK and America, but not for the treatment of JIA-Uveitis. If the trial showed that adalimumab was effective in controlling the inflammation in this severe eye complication of JIA, it could hopefully decrease the proportion of children developing serious visual complications and blindness from uncontrolled uveitis.

In fact, the study team proved the new treatment to be so successful that its trial was stopped early by the data safety monitoring committee, leading to NHS England’s immediate approval for access to the new drug across England. AbbVie Inc obtained license and market authorisation from the European Medicines Agency to enable access to the drug for thousands of children across Europe. This access may soon be replicated in the US, after Federal Drugs Agency approval.

In November 2017, Professor Ramanan’s successful trial design was recognised by the University when he and the Uveitis (Sycamore) Study team won the Vice Chancellor’s Impact Prize 2017 in Health and Wellbeing.